Corporate Presentation
September 2022
Fortress Biotech
Forward Looking Statements
This presentation may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section
21E of the Securities Exchange Act of 1934, as amended. For such forward-looking statements, we claim the protection of the safe harbor for forward-
looking statements contained in the Private Securities Litigation Reform Act of 1995. As used below and throughout this presentation, the words “we”,
“us” and “our” may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but
are not limited to, any statements relating to our growth strategy, products and product development programs and any other statements that are not
descriptions of fact. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that
could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially
from those currently anticipated include: risks related to our growth strategy; risks relating to the results of research and development activities; our
ability to obtain, perform under and maintain financing and strategic agreements and relationships; uncertainties relating to preclinical and clinical
testing; our dependence on third party suppliers; our ability to attract, integrate, and retain key personnel; the early stage of products under
development; our need for and continued access to additional funds; government regulation; patent and intellectual property matters; competition; as
well as other risks described in our Securities and Exchange Commission filings. We expressly disclaim any obligation or undertaking to release
publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in
events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is
intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this
presentation should be read as applying mutatis mutandis to every other instance of such information appearing herein. This presentation may
contain depictions of Fortress’ percentage ownership positions in several of its affiliated companies; while we endeavor to update such figures
regularly, these percentages are subject to periodic change for a variety of reasons, and updates may not occur more frequently than every calendar
month or quarter. Accordingly, you should understand that the percentage figures presented herein may only portray Fortress’ ownership positions as
of the most recent update, and not necessarily as of the date on which you are reviewing these materials.
2
Fortress has an extensive portfolio of commercial and development assets
and a business development engine for generating new opportunities
3
Portfolio and pipeline includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and at partner companies that Fortress may otherwise have an economic interest in
* Includes two trials at partner company Caelum Biosciences; AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone
payments. FBIO received ~$56.9 million of such upfront amount and is eligible to receive ~42% of the proceeds from all future milestone payments.
20 clinical-stage programs
30
ongoing clinical
trials
7*
ongoing pivotal /
registrational trials
9 marketed programs
2
NDA/BLA submissions
anticipated over next
six to twelve months
Preclinical and Early-to-Mid-Stage Clinical
Progression
Late-Stage and Regulatory Execution Commercial Scaling
$39.0M
Journey net sales (2022 H1)
77%
CAGR on net sales (2016-2021)
Over $150M spent on R&D activities across pipeline candidates by Fortress and our
partner companies in 2021
Commercial Late Clinical Early Clinical Preclinical
Qbrexza
®
CUTX-101 MB-102 AAV.sFH Gene Therapy
Targadox
®
Cosibelimab MB-106 AAV-ATP7A Gene Therapy
Ximino
®
Olafertinib Triplex ConVax
Exelderm
®
CAEL-101
**
MB-101 CEVA-102
AMZEEQ
®
MB-107 MB-108 CEVA-D
ZILXI
®
MB-207 MB-109 CK-103
9 Total Marketed
Dermatology Products
*
DFD-29 MB-103 CK-302
CEVA-101 MB-104 CK-303
MB-105 In vivo CAR T Technology
MB-110
BAER-101
Dotinurad
Fortress product portfolio by stage
4
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and at partner companies that Fortress may otherwise have an economic interest in
*
9 total products includes Qbrexza, Targadox, Ximino, Exelderm, Amzeeq, and Zilxi
**AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such upfront
amount and is eligible to receive ~42% of the proceeds from all future milestone payments.
Therapeutic Areas/Modalities
Dermatology Rare Disease Rheumatology
Oncology / Hematology Other
Our strategy is focused on finding and developing lower risk opportunities
efficiently, which creates inherent advantages
5
Key Success Drivers
Deep pharmaceutical
development experience
across our team and network
of leading advisors
Focused BD search
and evaluation criteria
that consistently finds assets
with high unmet need
and balanced clinical /
development risk
Lower risk investment
and development strategy
due to diverse pipeline
across therapeutic area,
modality, stage and number
of shots on goal
Proven track record
in licensing attractive
therapies fast and first,
which avoids excessively
competitive processes
Differentiated business
model provides synergistic
benefits of scale and
increased opportunity
Fortress pipeline and structure presents a de-risking strategy with
diversified exposure and many shots on goal
6
Preclinical
Late-Stage and Regulatory
Execution
Commercial Scaling
Fortress Asset Pipeline
Therapeutic Areas/Modalities
Dermatology Rare Disease Rheumatology
Oncology / Hematology Other
Early-to-Mid-Stage Clinical
Progression
Diversified portfolio with programs in
multiple therapeutic areas across all
development and commercial stages
‒ Approach reduces risk to portfolio building versus
single asset/platform competitors
‒ Exposure to various technologies and therapeutic
areas, all with significant upside potential
14K + Clinical-stage
Asset Universe
Core expertise in quickly identifying and in-licensing/acquiring de-risked
therapeutics, creating a continuous BD engine
7
Fortress NewCo
Fortress Existing
Partner Company
High unmet need for
patients
Proof-of-concept data or
validated mechanism/pathway
Capital efficient
development path
Market opportunity
Fortress Portfolio
8
Fortress develops and markets therapeutic products through a portfolio of
majority-owned and controlled companies
I/O & Targeted Oncology Menkes Disease
CAR-T Cell Therapy
& Gene Therapy
Dermatology
Gene Therapy:
Dry AMD, aHUS
Immunotherapy:
Cytomegalovirus
Cell Therapy:
Traumatic Brain Injury
CNS Disorders
• Each subsidiary/partner company is focused on clinical and commercial execution of their products with support from Fortress
• Partner company format allows for flexibility to pursue deals, partnerships and fundraising
• Each partner company provides multiple ways to create meaningful streams of revenue and equity
Rheumatology:
Gout
Fortress and our partner companies share mutually beneficial relationships
9
Benefits of Fortress and Subsidiary/Partner Company
Relationship
• Fortress provides all business development efforts including
active identification of synergistic portfolio assets
• Fortress supports with ongoing operational, strategic,
administrative and finance support
• Most subsidiary/partner companies provide Fortress an annual
2.5% equity dividend and a 4.5% royalty on net sales*, which
incentivizes Fortress to continue to build value over time
• Subsidiary/partner companies and Fortress can share resources,
personnel, and expertise
*Applicable to most Fortress subsidiaries, but not all
Asset
Monetizations
Fortress share of
distributions from PRV
sales* and asset
monetizations
Upon FDA approval,
Sentynl to acquire CUTX-101
and pay to Cyprium $20M,
sales milestones up to $255M,
and royalties^ + PRV monetization
of ~$100M-$110M
(Fortress owns ~70% of Cyprium)
10
Shareholder value is generated through asset monetizations, equity
holdings/appreciation, and equity dividend and royalty revenues
+
*Priority Review Vouchers (PRV) are granted by the FDA for certain rare pediatric disease approvals and can be transferred between companies (recent PRVs have sold for $100M-$110M)
**10% of upfront payment held in escrow to satisfy potential indemnification obligations, if any
***Applicable to most Fortress subsidiaries, but not all
^ Subject to potential reductions as per Sentynl Agreement, refer to Footnote regarding CUTX-101 on Slide 12
Fortress Biotech Value Drivers
+ =
Equity
Holdings
Fortress holds a large
stake of equity in each
subsidiary company and
shareholders gain value
during appreciation of
equity or through exits
AstraZeneca acquired Caelum
for an upfront payment of ~$150M**
and additional potential payments
totaling up to $350M payable
to Caelum Shareholders
(Fortress eligible to receive ~42%)
Dividend &
Royalty Revenue
Subsidiary companies***
pay to Fortress an
annual equity dividend
worth 2.5% of subsidiary
market cap and a 4.5%
royalty on net sales
Increasing
Shareholder
Value
Building shareholder value through long-term growth and scale
11
Product
Revenue
Royalties
Equity
Holdings
Equity
Dividends
Fortress’ business model has the potential for
“snowballing” growth as we acquire new assets and as
our subsidiary/partner companies grow in value
12
Potential revenue and cashflow events
= Anticipated product revenue/royalties
= Potential PRV/milestone/monetization proceeds
= Potential regulatory approval
Approval, data, and trial timings are internal estimates based on current knowledge and potential timelines are subject to change. Potential peak sales are based on internal forecasts and/or market comps and assume approval in all
denoted indications and are subject to change; * Asset is currently fully controlled by AstraZeneca through the acquisition of Caelum, all estimates and dates related to CAEL-101 are based on Fortress estimates and not guidance
provided by Caelum or AstraZeneca. Fortress remains eligible to receive up to an additional ~$155 million in escrow release and milestone payments from the transaction;
^ Cyprium is currently in a dispute with its contract manufacturing organization (the “CMO”), regarding the CMO’s attempt to terminate a Master Services Agreement (“MSA”) between Cyprium and the CMO. Cyprium believes the
CMO’s grounds for purporting to terminate the MSA are without merit and is currently availing itself of all appropriate legal remedies in efforts to ensure that the CMO abides by its obligations under the MSA. To that end, Cyprium
obtained a temporary restraining order on August 8, 2022, requiring the CMO to provide at least 72 hours prior written notice to Cyprium before taking any action that may prevent the CMO from performing its obligations under the MSA
pending Cyprium’s application for a preliminary injunction. Cyprium’s timeline for completing submission of its NDA application for CUTX-101 may be delayed during the pendency, and following resolution, of such dispute.
< $500M $500M - $1B
> $1B
✓
Partner
Company
Asset(s) 2020 2021 2022 2023 2024 2025 2026+
Potential Peak Sales
(Global)
Journey
(DERM)
Commercial Portfolio
Qbrexza, Ximino, Targadox, etc.
$44.5M
Net Sales
$63.1M
Net Sales
$39.0M
H1 Net Sales
DFD
-29 (oral small molecule)
Two Phase 3s for rosacea
Caelum*
CAEL
-101 (fibril-reactive mAb)
Pivotal trial for AL amyloidosis
$56.9M
Monetization
Checkpoint
(CKPT)
Cosibelimab (anti
-PD-L1 mAb)
BLA submission for metastatic and
locally advanced
cSCC
Olafertinib
(3
rd
gen EGFRi)
Phase 3 trial in Asia in NSCLC (partner)
(Asia)
Cyprium^
CUTX
-101 (copper histidinate)
NDA submission for Menkes Disease
$8M
Monetization
Mustang
(MBIO)
MB
-107 (gene therapy)
Pivotal Phase 2 trial for newly diagnosed
XSCID planned 2023
MB
-207 (gene therapy)
Pivotal Phase 2 trial for previously
transplanted XSCID planned 2023
MB
-109 (CAR-T + oncolytic virus)
IND filing for GBM planned 2023
MB
-106 (CD20 CAR-T)
Phase 1/2 trial for NHL & CLL
Urica
Dotinurad (URAT1 inhibitor)
Phase 1 trial ongoing
✓
✓
✓
✓
✓
Potential near-term value-creating events for Fortress shareholders
13
CUTX-101, DFD-29, MB-106, MB-107, MB-109, MB-207, Cosibelimab, Olafertinib, and Dotinurad are product candidates in development at Fortress subsidiary/partner companies
* CAEL-101 is currently fully controlled by AstraZeneca through the acquisition of Caelum, all estimates and dates related to CAEL-101 are based on Fortress estimates and not guidance provided by Caelum or AstraZeneca
Approval, data, and trial timings are internal estimates based on current knowledge and potential timelines are subject to change
^ Refer to Footnote regarding CUTX-101 on Slide 12
+
Urica Therapeutics, Inc. renamed from UR-1 Therapeutics, Inc.
Category Company Asset
Anticipated Milestone Anticipated Timing
Monetization
Events
Cyprium^
CUTX-101
o Potential milestone payments and PRV sale 2023
o Eligible to receive ~70% of up to $267M in remaining regulatory and sales milestones 2023+
Caelum*
CAEL-101
o Eligible to receive ~42% of additional potential payments to Caelum shareholders totaling up
to $350M from regulatory and commercial milestones
2023+
Regulatory
Decisions
Cyprium^
CUTX-101
o FDA Decision for Menkes Disease 2023
Checkpoint
Cosibelimab
o FDA Decision for metastatic and locally advanced cSCC 2023
Caelum*
CAEL-101
o FDA Decision for AL Amyloidosis 2023+
Clinical
Data
Urica
+
Dotinurad
o Phase 1 data readout 2H 2022
Mustang
MB-106
o Potential Phase 1 data readout from both FHCRC & Mustang clinical trials 4Q 2022
Journey
DFD-29
o Anticipate readouts of two Phase 3 clinicals trials for rosacea 1H 2023
Checkpoint
Olafertinib
o Potential Phase 3 data readout in NSCLC in Asia 2023+
Caelum*
CAEL-101
o Potential readout of the CAELUM CARES Phase 3 2023+
Trial Initiation Mustang
MB-106
o Dosing of first patient in Mustang IND Phase 1 trial in NHL/CLL 3Q 2022
MB-107
o Initiation of pivotal trial for XSCID in newly diagnosed patients 2023
MB-207
o Initiation of pivotal trial for XSCID in previously transplanted patients 2023
MB-109
o Initiation of novel combination (CAR-T + oncolytic virus) trial for GBM 2023
MB-106
o Initiation of Phase 2 pivotal study in NHL/CLL 2023
Recent/near-term monetization opportunities
14
*Proceeds are net of transaction expenses and escrow
Blue text represents proceeds received already by respective company and Fortress
^ Refer to Footnote regarding CUTX-101 on Slide 12
Cyprium^ Development & Asset
Purchase Agreement with Sentynl
February 2021
Caelum Acquired by AstraZeneca
October 2021
• Upon FDA approval, Sentynl to acquire CUTX-101 for up to
$20M in upfront and regulatory milestone payments through
NDA approval – $8M was paid upon execution of the
agreement in February 2021
• Cyprium eligible to receive sales milestones up to $255M
and royalties on CUTX-101 net sales are also payable:
– 6% due on portion of annual net sales up to $75M
– 17.5% due on portion of annual net sales between $75M and
$100M
– 25% due on portion of annual net sales over $100M
• Cyprium will retain 100% ownership over any FDA PRV that
may be issued at NDA approval for CUTX-101. Recent data
suggests PRVs may be worth $100M to $110M
• Option exercise triggered upfront payment of
approximately $150M to Caelum shareholders, of which
$56.9M* was payable to Fortress
• Additional potential payments to Caelum shareholders
totaling up to $350M, payable upon the achievement of
regulatory and commercial milestones
• AstraZeneca intends to advance and accelerate the Phase 3
development of CAEL-101 for light chain (AL) amyloidosis
• Fortress is eligible for up to a total of $212M in proceeds
(~42%) from this transaction
$75 million debt
financing closed with
Runway Growth Capital
Fortress found the
CD20 CAR-T at FHCRC*
Fortress enabled Mustang’s $94.5 million
private placement financing and buildout
of cell processing facility
Fortress found two CAR-T
assets from City of Hope and
founded Mustang Bio
Fortress identified an ex-vivo
lentiviral gene therapy asset
from St. Jude and NIH
Fortress identified an
oncolytic virus (C134) at
Nationwide Children’s
Fortress found a preclinical in vivo CAR-T
asset (Mayo Clinic) and Phase 1/2 RAG1-SCID
asset (Leiden University Medical Center)
15
Assets In-Licensed
To Mustang Portfolio
Fortress Value Add +
Key Events
Mustang Bio
Founded
Two CAR-T assets
(MB-101 & MB-102)
CD20 CAR-T
(MB-106)
LVV gene therapy
(MB-107/MB-207)
C134 Oncolytic Virus
(MB-108)
*Fred Hutchinson Cancer Research Center (FHCRC)
in vivo CAR-T technology and
RAG1 program (MB-110)
Fortress has continuing equity and royalty interests in Mustang, incentivizing us to keep building value in Mustang
2015 2016 2017 2018 2019 2020 2021
Adding more horsepower to Mustang over time
2022
16
Caelum Biosciences – acquired by AstraZeneca in Oct 2021
Fortress and Caelum complete a third-party
financing of Convertible Notes raising $9.9M
3Q 2017
Caelum announces positive Phase 1a/1b data, which
demonstrated that CAEL-101 improved organ function
in AL amyloidosis
December 2017
Caelum initiates Phase 2 study
of CAEL-101 in AL Amyloidosis
March 2020
Caelum initiates Phase 3 studies
of CAEL-101 in AL Amyloidosis
September 2020
Agreement includes additional potential payments
totaling up to $350M payable to Caelum Shareholders
upon the achievement of regulatory and commercial
milestones (Fortress eligible to receive ~42%)
2022 & Beyond
January 2017
October 2021
January 2019
Caelum
Founded
Caelum
Acquired
R&D Funding
Agreement
Fortress found a novel antibody at Columbia University
being evaluated in a Phase 1a/1b study with
promising interim data and licensed the asset
AstraZeneca acquired Caelum for an upfront payment
of ~$150M, of which ~$63M payable to Fortress
including ~$6M in escrow
Caelum entered into a strategic agreement with Alexion
Pharmaceuticals to advance CAEL-101 where Alexion
would pay up to $60M for continued development/equity
and have an exclusive option to acquire Caelum
Note: AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such upfront
amount (with $6 million in escrow) and is eligible to receive ~42% of the proceeds from all future milestone payments.
17
$3.6
$15.5
$23.4
$34.9
$44.5
$63.1
$0.0
$10.0
$20.0
$30.0
$40.0
$50.0
$60.0
$70.0
2016
2017
2018
2019
2020
2021
Journey Product Net Sales (2016 – 2021)
$ in millions
*CAGR for net sales growth from 2016-2021
Journey of constant growth and portfolio expansion
Launched / acquired 3 new
products in 2021, including Phase 3
asset DFD-29 for rosacea
Record $63.1M in 2021 sales
and $39M in H1’22 revenue
Acquired 2 new commercial
products from Vyne Therapeutics in
January 2022 (AMZEEQ and ZILXI)
Pipeline Detail
18
19
Late-Stage Portfolio – Multiple near-term value inflection points
Candidate Indication(s) Phase 1 Phase 2
Pivotal /
Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
CUTX-101^
Copper Histidinate
Menkes Disease
Rolling NDA submission in process^
70% of Cyprium
4.5% Royalty
2.5% Equity Dividend
COSIBELIMAB
Anti-PD-L1 mAb
Recurrent
or metastatic cancers
Planned BLA submission end of year in metastatic
and locally advanced cSCC
20% of CKPT
4.5% Royalty
2.5% Equity Dividend
OLAFERTINIB
Mut.-EGFR Inh.
EGFR
+
NSCLC
Phase 3 study ongoing by partner
CAEL-101
mAb 11-1F4
Light chain (AL)
amyloidosis
Acquired by AstraZeneca in Oct 2021
Two ongoing global Phase 3 studies for AL
amyloidosis
42% of future proceeds
to Caelum from
AstraZeneca**
$56.9M received***
~$150M in potential
future proceeds
MB-107
Gene Therapy
XSCID
(newly diagnosed)
Anticipate dosing first patient in MB-107
registrational trial in 2023
19% of MBIO
4.5% Royalty
2.5% Equity Dividend
MB-207
Gene Therapy
XSCID
(previously transplanted)
Anticipate dosing first patient in MB-207
registrational trial in 2023
DFD-29
Oral Small Molecule
Rosacea
Topline data from Phase 3 program in rosacea
expected 1H 2023
58% of DERM
CEVA-101
Cell Therapy
Traumatic brain injury
(pediatric and adult)
Phase 2 study in Peds completed 1H 2021
Phase 2 data in Adults expected 2023
78% of Cellvation
4.5% Royalty
2.5% Equity Dividend
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
**AstraZeneca’s Alexion acquired Caelum Biosciences on 10/5/2021 for up to $500 million, including $150 million upfront and up to $350 million in future contingent milestone payments. FBIO received ~$56.9 million of such
upfront amount and is eligible to receive ~42% of the proceeds from all future milestone payments.
***Net of transaction expenses and escrow
^
Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Jan 1, 2022
^ Refer to Footnote regarding CUTX-101 on Slide 12
< $500M $500M - $1B
> $1B
20
Early-to-Mid Stage Portfolio – Expansive pipeline for high unmet need areas
Candidate Indication(s) Preclinical Phase 1 Phase 2 Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
MB-102
CD123 CAR-T
Blastic plasmacytoid
dendritic cell neoplasm
Mustang IND trial enrolling
19% of MBIO
4.5% Royalty
2.5% Equity Dividend
MB-106
CD20 CAR-T
B-Cell Non-Hodgkin
Lymphoma, and CLL
Anticipate dosing of first patient in Mustang IND
trial in 3Q 2022 and first disclosure of data from
Mustang-IND trial in 4Q 2022
Triplex
Vaccine
Cytomegalovirus (CMV)
Initiated HIV/CMV co-infection Phase 2 trial;
received NIAID/NIH grant of potentially more than
$20M to fund Phase 2 study in liver transplant
82% of Helocyte
4.5% Royalty
2.5% Equity Dividend
MB-101
IL13Rα2 CAR-T
Recurrent glioblastoma
(GBM)
File IND for Phase 1 combination trial for MB-109
(MB-101 + MB-108) in 2023
19% of MBIO
4.5% Royalty
2.5% Equity Dividend
MB-108
Oncolytic Virus (OV)
Recurrent GBM
MB-109
IL13Rα2 CAR-T + OV
Recurrent GBM and
anaplastic astrocytoma
MB-103
HER2 CAR-T
GBM and Metastatic
Breast Cancer to Brain
Data disclosure from COH Phase 1 trials expected
MB-104
CS1 CAR-T
Multiple Myeloma (MM)
Data disclosure from COH Phase 1 trial expected
MB-105
PSCA CAR-T
Prostate & Pancreatic
Cancers
Next data disclosure from COH Phase 1 prostate
cancer trial expected 1H 2023
MB-110
Gene Therapy
RAG1-SCID
Ongoing Phase 1/2 multi-center trial in Europe
BAER-101
α2/3–subtype-GABA A
PAM
CNS Disorders
Preclinical POC data to support IND in Refractory
Epilepsy anticipated 2022
61% of Baergic
4.5% Royalty
2.5% Equity Dividend
Dotinurad
URAT1 inhibitor
Gout and Chronic Kidney
Disease
Phase 1 data expected H2 2022
65% of Urica
Therapeutics
4.5% Royalty
2.5% Equity Dividend
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
^
Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Jan 1, 2022
< $500M $500M - $1B
> $1B
21
Preclinical Portfolio – Long-term value potential for key therapeutic areas
Candidate Indication(s) Preclinical Phase 1 Phase 2 Phase 3
Status / Upcoming Anticipated Milestones
FBIO Ownership % /
Royalty
†
Potential Peak
Sales (Global)
^
AAV.sFH
AAV Gene Therapy
Dry AMD and aHUS
Non-human primate (NHP) long-term toxicology
data and additional POC in Dry AMD
54% of Aevitas
4.5% Royalty
2.5% Equity Dividend
AAV-ATP7A
AAV Gene Therapy
Menkes Disease
Nominate candidate for clinical development
70% of Cyprium
4.5% Royalty
2.5% Equity Dividend
CK-103
BET Inhibitor
Solid Tumors
Potential Phase 1 initiation
20% of CKPT
4.5% Royalty
2.5% Equity Dividend
ConVax
Vaccine
CMV Prevention & Control
IND filing
82% of Helocyte
4.5% Royalty
2.5% Equity Dividend
CEVA-102
Cell Therapy
TBI, GvHD, ARDS, CHF,
Crohn’s (Off-the-Shelf)
IND filing
78% of Cellvation
4.5% Royalty
2.5% Equity Dividend
CEVA-D
Bioreactor Device
Mechano-transduction
Device for Cell Therapies
Data published in Frontiers in Immunology (July
2022)
In vivo CAR-T
Off-the-shelf CAR-T
Platform
Data publication in 2023
19% of MBIO
4.5% Royalty
2.5% Equity Dividend
Portfolio includes product candidates in development at Fortress, at its majority-owned and majority-controlled partners, and partner companies that Fortress may otherwise have an economic interest in.
^
Potential peak sales are based on internal forecasts and/or market comps and assume approval in all denoted indications and are subject to change
†
Ownership estimated as of Jan 1, 2022
< $500M $500M - $1B
> $1B
22
Cosibelimab
Anti-PD-L1 mAb for treatment of metastatic and
locally advanced cSCC
Asset Overview
• Fully human IgG1 monoclonal antibody that is potentially
differentiated versus marketed PD-(L)1s
• ASCO 2022 presentation of top-line data in metastatic cSCC
(n=78) showed a confirmed objective response rate (ORR) by
independent central review in the modified intent to treat
population of 48.7% and 13.2% of patients achieving a
complete response in target lesions.
• In locally advanced cSCC, as of March 2022 data cutoff,
confirmed ORR by independent central review in 31 patients
was 54.8%.
• Composition of Matter patent issued in US, expiring no earlier
than 2038
Est. Market
-L1 mAbs: $40B+/year
Status
Next Steps
Near
-Term
Value Creation
-term value creation from clinical
Product candidate in development at Checkpoint Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still
maintains a large ownership position.
23
CUTX-101
Subcutaneous injectable formulation of Copper
Histidinate for patients with Menkes Disease
Asset Overview
• Reported positive top-line clinical efficacy data, showing a
nearly 80% reduction in the risk of death (Hazard Ratio = 0.21,
p<0.0001)
Monetization Overview
• Upon FDA approval, Sentynl to acquire CUTX-101 for up to
$20M in upfront and regulatory milestone payments through
NDA approval – $8M was paid upon execution of the
agreement in February 2021
• Cyprium eligible to receive sales milestones totaling up to
$255M and tiered royalties (6% of net sales up to $75M, 17.5%
between $75M and $100M, 25% over $100M)
• Cyprium will retain 100% ownership over any FDA PRV that
may be issued at NDA approval for CUTX-101**
• FBIO owns ~70% of Cyprium
Est. Market
Estimated 50
-225 patients per year in the US
1
alone
with Menkes with potential for PRV worth
approximately $100M
-110M**
Status
Phase 3 complete; ongoing rolling submission of
NDA to FDA
Next Steps
Complete rolling submission of NDA^
Near
-Term
Value Creation
Potential near
-term value creation from milestones,
royalties, and PRV monetization
Product candidate in development at Cyprium Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still maintains a
majority ownership position.
1
Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome
Aggregation Database (gnomAD). Mol Genet Metab Rep. 2020;24:100602.
**In the event of a sale of a PRV by Cyprium, Cyprium would be obligated to make payments to the NIH and to holders of Cyprium’s 9.375%
Series A Cumulative Redeemable Perpetual Preferred (all as disclosed in Fortress’ public filings).
^ Cyprium is currently in a dispute with its contract manufacturing organization (the “CMO”), regarding the CMO’s attempt to terminate a Master
Services Agreement (“MSA”) between Cyprium and the CMO. Cyprium believes the CMO’s grounds for purporting to terminate the MSA are
without merit and is currently availing itself of all appropriate legal remedies in efforts to ensure that the CMO abides by its obligations under the
MSA. To that end, Cyprium obtained a temporary restraining order on August 8, 2022, requiring the CMO to provide at least 72 hours prior
written notice to Cyprium before taking any action that may prevent the CMO from performing its obligations under the MSA pending Cyprium’s
application for a preliminary injunction. Cyprium’s timeline for completing submission of its NDA application for CUTX-101 may be delayed during
the pendency, and following resolution, of such dispute.
24
Dotinurad
URAT1 inhibitor for gout, chronic kidney disease,
and heart failure
Asset Overview
• Potential to be the most potent oral therapy for lowering serum
uric acid levels (sUA) with excellent safety profile
• Over 1000 patients treated in clinical trials
• Improved selectivity profile versus other uricosurics with
extensive data in humans showing excellent efficacy and safety
profile (>500 patients in Japan Phase 3 trials treated for up to
58 weeks)
• Dotinurad (URECE
®
tablet) was approved in Japan in 2020 as a
once-daily 1
st
line oral therapy for gout and hyperuricemia
• Unique product design and positioning that address key unmet
needs in US gout treatment paradigm
Est. Market
-3 million refractory gout patients and >10
Status
Next Steps
Near
-Term
Value Creation
-term value creation from clinical
Product candidate in development at Urica Therapeutics, Inc., an entity which was founded by Fortress and in which Fortress still maintains a
majority ownership position.
FUJI YAKUHIN
25
MB-106
CD20 Autologous CAR-T Cell Therapy for NHL and
CLL
Asset Overview
• Third generation fully-human CD20 targeted autologous CAR-T
• FDA granted Orphan Drug Designation for Waldenstrom
macroglobulinemia (WM) in June 2022
• Update from Phase 1/2 trial presented at EHA 2022
– Overall Response Rate (ORR) of 96% and Complete Response (CR)
rate of 73% was observed (n=26) across all dose levels
– Durable responses observed in wide range of hematologic
malignancies including FL, CLL, DLBCL, and WM
– Two patients that had prior CD19-directed CAR T therapy
experienced CRs following MB-106 therapy (one patient with FL and
one patient DLBCL)
– 9 patients remain in CR for greater than 1 year
– Favorable safety profile was observed in all patients, with no grade 3
or 4 cytokine release syndrome or immune effector cell‐associated
neurotoxicity syndrome (ICANS), and none of the FL patients
experienced ICANS of any grade (n=18)
Est. Market
Status
Next Steps
-IND Phase 1 trial and multicenter
-IND Phase 1 trial
Data
-
-IND trial by the end of 2022; Potential
Macroglobulinemia in 4Q 2022
Near
-Term
Value Creation
-term value creation from clinical
Product candidate in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
26
MB-109
IL13Rα2 CAR-T + HSV-1 Oncolytic Virus for
Glioblastoma Multiforme
Asset Overview
• MB-101 + MB-108 combination is designed to turn the tumor
microenvironment “hot”, which may improve the efficacy of CAR-
T cell therapy
– MB-108 HSV-1 (herpes simplex virus 1) oncolytic virus infects tumor
cells, which reshapes the tumor microenvironment (TME) through
cytokine release and recruitment of endogenous CD8-positive
effector T cells
– “Hot” TME may enable MB-101 CAR T cells to better infiltrate the
tumor mass, undergo activation and effect tumor cell killing
Est. Market
Status
-101 (IL13Rα2 CAR-T) Phase 1 complete and
-108 (HSV-
Next Steps
-109
-101 + MB-108) in 2023
Near
-Term
Value Creation
-term value creation from clinical
Product candidate in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
27
MB-107 & MB-207
Ex-vivo lentiviral vector gene therapy for XSCID
“Bubble Boy” Disease
Asset Overview
• MB-107 for treatment of newborn XSCID patients and MB-207
for treatment of previously transplanted XSCID patients
• MB-107 published clinical results demonstrated**:
– Broad immune reconstitution with excellent safety profile
– 100% T cell reconstitution with no loss of immunity
– Up to 5 years of follow up for patients
• MB-207 published clinical results demonstrated***:
– T cell reconstitution achieved in all patients; no loss of immunity
• No insertional mutagenesis observed in either program
FDA and EMA Designations
Est. Market
107: XSCID incidence of ~1 in 225K newborns per
year (worldwide)
207: ~400 patients living with XSCID post
-
transplant
in the US and ~650 patients ex
-US high/mid-
income
markets
Status
Entering pivotal multi
-center Phase 2 trials
Next Steps
MB
-107: First patient dosing in Phase 2 reg. trial in
newly diagnosed patients expected 2023
MB
-207: First patient dosing in Phase 2 reg. trial in
previously transplanted patients expected 2023
Near
-Term
Value Creation
Potential near
-term value creation from clinical
development progression, potential approvals/PRV
monetizations
^
and MBIO equity appreciation
Product candidates in development at Mustang Bio, Inc., an entity which was founded by Fortress and in which Fortress still maintains a large
ownership position.
** ASGCT 2022 update by Mamcarz E, et al
***De Ravin SS et al. Blood (2019) 134 (Suppl 1): 608.
^ Recent data suggests PRVs may be worth ~$100M to ~$110M, both MB-107 and MB-207 are each eligible for PRVs
MB-107 MB-207
FDA Designations
RMAT
✓
(requesting in 2023)
Rare Pediatric Disease
✓ ✓
Orphan Drug
✓ ✓
EMA Designations
Orphan Drug
✓ ✓
ATMP
✓ ✓
PRIME
✓
28
CAEL-101*
Monoclonal antibody (mAb) for the treatment of
patients with amyloid light chain (“AL”) amyloidosis
Asset Overview
• Granted Orphan Drug designations in the U.S. and EU
• No FDA, EMEA, or PMDA approved therapies for indication
• Potentially understated market size given AL Amyloidosis often
misdiagnosed
Monetization Overview
• The agreement triggered upfront payment of approximately
$150M to Caelum shareholders (of which approximately
$56.9M** was payable to Fortress Biotech) and provides for
additional potential payments to Caelum shareholders totaling
up to $350M upon the achievement of regulatory and
commercial milestones
• Fortress is eligible to receive approximately 42% of all proceeds
from the transaction
*As Caelum was acquired by AstraZeneca in 2021, Fortress may not be apprised of ongoing developments pertaining to CAEL-101 to the same
degree that Fortress had been prior to such acquisition; accordingly, the information presented on this slide may not reflect the latest disposition
of the product candidate
**In each case, figures are net of transaction expenses and escrow.
Est. Market
-diagnosed patients (U.S.) per year
Status
Next Steps
Near
-Term
Value Creation
-term
Top-tier academic & commercial partners
29
FUJI YAKUHIN
THANK YOU!